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Enzyme Replacement Therapy
ELAPRASE + Prophylactic Therapy for Hunter Syndrome
Phase 4
Recruiting
Research Sponsored by Takeda
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participant must have a documented diagnosis of MPS II
Participant has a deficiency in iduronate-2-sulfatase (I2S) enzyme activity of <= 10% of the lower limit of the normal range as measured in plasma, fibroblasts, or leukocytes
Timeline
Screening 3 weeks
Treatment Varies
Follow Up every 6 months up to 24 months
Awards & highlights
Study Summary
This trial investigates if a prophylactic therapy can prevent or reduce high titer antibodies in Hunter syndrome patients receiving ELAPRASE for up to 104 weeks.
Who is the study for?
This trial is for treatment-naïve boys under 6 with Hunter syndrome, having specific enzyme deficiencies and gene mutations. They must not have used ELAPRASE or certain other treatments recently and should be able to follow the study protocol without any medical conditions that could interfere.Check my eligibility
What is being tested?
The study tests if a preventive immune regimen can stop or lessen high titer anti-idursulfase antibodies in patients taking ELAPRASE. It includes Rituximab, Methotrexate, IVIG alongside ELAPRASE over up to 104 weeks with assessments every six months.See study design
What are the potential side effects?
Possible side effects include reactions related to the immune system such as infusion reactions from Rituximab or IVIG, liver issues due to Methotrexate, and general symptoms like fever, chills, nausea which may vary among individuals.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with MPS II.
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My enzyme (I2S) activity is very low, below 10% of the normal range.
Select...
I have a severe IDS gene mutation linked to a strong immune response to enzyme therapy.
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I am under 6 years old.
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I am male.
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I have never taken ELAPRASE before.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ every 6 months up to 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~every 6 months up to 24 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Rate of Anti-Idursulfase Antibodies Formation, Including Anti-Idursulfase Antibodies That Have Enzyme Neutralizing Activity
Secondary outcome measures
Change From Baseline in Liver Volume
Change From Baseline in Normalized uGAG per Upper Limit of Normal for age (uGAG)/ULN)
Glycosaminoglycans
+1 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: ITR + ELAPRASEExperimental Treatment4 Interventions
Participants will receive prophylactic ITR which consist of rituximab, methotrexate and IVIG in a 5-week cycle. Following the completion of 1 cycle and at the Month 6, 12, and 18 study visits, an assessment will be made regarding the need for administering another 5-week cycle of the ITR depending on the trend of the participants anti-idursulfase antibody titers and lymphocyte quantitation and CD19 percent (%) recovery.
Elaprase treatment (IV, weekly) will start 1 day after the initiation of the first cycle of ITR and continue for 104 weeks.
The dose of ELAPRASE will be calculated based on the participant's weight at each visit.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Rituximab
1999
Completed Phase 4
~1880
Methotrexate
2013
Completed Phase 4
~3800
Find a Location
Who is running the clinical trial?
TakedaLead Sponsor
1,203 Previous Clinical Trials
4,177,939 Total Patients Enrolled
4 Trials studying Hunter Syndrome
34 Patients Enrolled for Hunter Syndrome
Takeda Development Center Americas, Inc.Industry Sponsor
56 Previous Clinical Trials
12,029 Total Patients Enrolled
1 Trials studying Hunter Syndrome
21 Patients Enrolled for Hunter Syndrome
Study DirectorStudy DirectorTakeda
1,211 Previous Clinical Trials
489,267 Total Patients Enrolled
14 Trials studying Hunter Syndrome
1,787 Patients Enrolled for Hunter Syndrome
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have been diagnosed with MPS II.Your enzyme activity levels for certain substances in your body are within the normal range.You do not have antibodies against the drug idursulfase in your blood.I am not on any medication that could interfere with the study.My enzyme (I2S) activity is very low, below 10% of the normal range.I do not have any health conditions that could interfere with the study or pose a risk.I have a condition that might interfere with the study drug's effects.I have a severe IDS gene mutation linked to a strong immune response to enzyme therapy.I have received a blood transfusion in the last 90 days.I am being treated with idursulfase-IT.I am under 6 years old.I am male.I have never taken ELAPRASE before.I have received growth hormones, a cord blood infusion, or a bone marrow transplant.
Research Study Groups:
This trial has the following groups:- Group 1: ITR + ELAPRASE
Awards:
This trial has 3 awards, including:- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What potential dangers are associated with ITR + ELAPRASE treatment?
"The safety of ITR + ELAPRASE was rated at 3, since Phase 4 trials demonstrate its already approved status."
Answered by AI
How many medical facilities are participating in this research project?
"This clinical trial is taking place at locations such as Children's Hospital and Research Center in Oakland, California; UC Davis Medical Centre in Sacramento, Illinois ; Rady Childrens Hospital San Diego - PIN in San Diego, Minnesota; plus 11 other sites."
Answered by AI
Is recruitment for this experiment still ongoing?
"As indicated on clinicaltrials.gov this research effort is currently recruiting participants, with the trial having been initially posted on February 28th 2023 and most recently updated on April 5th of that same year."
Answered by AI
How many participants has been recruited for this clinical trial?
"Takeda, the sponsoring body of this trial, needs 5 suitable candidates that meet its inclusion criteria and will be running it from Children's Hospital and Research Center at Oakland in Oakland, California as well UC Davis Medical Centre in Sacramento."
Answered by AI
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